sepiapterin
Trade name: Sephience
Small moleculeapprovedOrphan Drug FDA
Approved
Jul 28, 2025
Sepiapterin, sold under the brand name Sephience, is a medication used for the treatment of hyperphenylalaninemia. Sepiapterin is a phenylalanine hydroxylase activator. — Wikipedia
Clinical trial activity
5 trials · 2 clinical orgs · 1 marketing orgs
Phase 1
1
Phase 2
5
Phase 3
3
Phase 4
0
Earliest trial started Jun 24, 2018 (NCT03519711)
Timeline
2020s
- Jan 1, 2021
PTC Therapeutics — Earliest Phase 3 Sponsor(trial)
- Mar 4, 2021
Orphan Drug Designation
- Jul 28, 2025
Earliest FDA Approval
- Jul 28, 2025
PTC Therapeutics — Marketing Organization
Indications
Approved for
Mechanism of action
- Unspecified targetUnclear
unclear
Approval history
- approvedJul 28, 2025
Chemistry & pharmacology
Loading structure…
SMILES
C[C@H](O)C(=O)C1=Nc2c(nc(N)[nH]c2=O)NC1- Mol. weight
- 237.22 g/mol
- Lipinski Ro5
- Pass
- Rule of 3
- No
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
Oral
No
Parenteral
No
Topical
No
Sources
- WikipediaSepiapterin ↗
- ChEMBLCHEMBL1255653 ↗
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT06302348 | Phase 3 | Mar 4, 2024 | PTC Therapeutics |
| NCT05166161 | Phase 3 | Jan 28, 2022 | PTC Therapeutics |
| NCT05099640 | Phase 3 | Sep 30, 2021 | PTC Therapeutics |
| NCT03712124 | Phase 2 | Feb 27, 2019 | Censa Pharmaceuticals |
| NCT03519711 | Phase 1/Phase 2 (Phase 2) | Jun 24, 2018 | Censa Pharmaceuticals |
Showing 5 of 5 trials
Page 1 / 1
Organizations
Research & Development (2)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| PTC Therapeutics | For profit | 3 | 3 | 1 | 2021 |
| Censa Pharmaceuticals | For profit | 2 | 2 | 1 | 2018 |
2 organizations
Page 1 / 1
Marketing (1)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| PTC Therapeutics | For profit | MKTG | Jul 28, 2025 |